A Study Evaluating the Safety, Tolerability, and Range of Biologically Active Doses of ICM-203 in Mild to Moderate Knee Osteoarthritis

NCT04875754 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: ICM 20-1001
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this study is to determine the safety, tolerability, and activity of ICM-203, a recombinant adeno-associated viral (AAV) vector that expresses a therapeutic gene that promotes cartilage formation, reduces joint inflammation and pain, as well as improves joint physical function, by injecting escalating doses of ICM-203 or matching placebo into the knee of subjects with mild to moderate knee osteoarthritis (OA).

Conditions

Osteoarthritis, Knee

Keywords

adeno-associated virus; gene therapy; ICM-203; osteoarthritis

Outcome Measures

Primary Outcomes (2)

• Treatment-Emergent Adverse Events (TEAEs)

  Incidence of Treatment-Emergent Adverse Events following administration of study drug

  Up to Week 52

• Severity of Treatment-Emergent Adverse Events (TEAEs)

  Severity of Treatment-Emergent Adverse Events graded according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0, following administration of study drug

  Up to Week 52

Secondary Outcomes (8)

• Knee pain

  Up to Week 52

• Knee function

  Up to Week 52

• Analgesic use

  Up to Week 52

• Magnetic Resonance Imaging Osteoarthritis Knee Score (MOAKS)

  Up to Week 52

• Joint space width

  Up to Week 52

Study Arms (3)

Group 1: ICM-203 (Low dose) vs Placebo

EXPERIMENTAL

8 subjects will be randomized to receive a single intra-articular injection of ICM-203 at 6x10e12 vg (n=6) or placebo (n=2) into the target knee at Day 1

Genetic: ICM-203; Drug: Placebo

Group 2: ICM-203 (Medium dose) vs Placebo

EXPERIMENTAL

4 subjects will be randomized to receive a single intra-articular injection of ICM-203 at 2x10e13 vg (n=3) or placebo (n=1) into the target knee at Day 1

Genetic: ICM-203; Drug: Placebo

Group 3: ICM-203 (High dose) vs Placebo (Optional)

EXPERIMENTAL

4 subjects will be randomized to receive a single intra-articular injection of ICM-203 at 6x10e13 vg (n=3) or placebo (n=1) into the target knee at Day 1

Genetic: ICM-203; Drug: Placebo

Interventions

ICM-203 GENETIC

Intra-articular injection

Group 1: ICM-203 (Low dose) vs Placebo; Group 2: ICM-203 (Medium dose) vs Placebo; Group 3: ICM-203 (High dose) vs Placebo (Optional)

Placebo DRUG

Intra-articular injection

Group 1: ICM-203 (Low dose) vs Placebo; Group 2: ICM-203 (Medium dose) vs Placebo; Group 3: ICM-203 (High dose) vs Placebo (Optional)

Eligibility Criteria

• Age: 50 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Body Mass Index (BMI) of 18.0 to 40.0 kg/m2, inclusive, at the time of screening.
• Kellgren-Lawrence grade 2 or grade 3 OA of target knee.
• Target knee pain between 4 and 9, inclusive, on an NRS ranging from 0 (no pain) to 10 (worst pain imaginable).
• KOOS function in daily living score \>25, a measure of knee function ranging from 0 (extreme problems) to 100 (no problems).
• A stable treatment regimen for symptomatic relief of OA, including NSAIDs, for 4 weeks prior to screening.

You may not qualify if:

• History of rheumatoid arthritis, psoriatic arthritis, gout, pseudogout, autoimmune OA, chondrocalcinosis, hemochromatosis, villonodular synovitis, and synovial chondromatosis or other disorder that in the opinion of the Investigator could cause inflammation of the knee.
• Injection of steroid, hyaluronate or other agent, into the target knee less than 90 days prior to day 1.
• Major injury to the target knee, such as torn ligament or severe sprain, within 12 months of screening.
• Disability so severe that the subject cannot comply with the study requirements, including knee symptoms that result in significant difficulty or inability to walk.
• Surgery on the target knee within 180 days prior to day 1
• Total knee arthroplasty or other knee surgery planned in the next 12 months.
• Active joint infection or other concurrent medical (diabetes, uncontrolled hypertension, severe osteoporosis, glaucoma) or psychiatric condition that, in the opinion of the Investigator, would make the subject unsuitable for the study.

Sponsors & Collaborators

• ICM Biotech Australia Pty Ltd.: lead

Study Sites (2)

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

Barwon Health

Geelong, Victoria, 3220, Australia

Location

MeSH Terms

Conditions

Osteoarthritis, Knee; Osteoarthritis

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Study Officials

• Alison Heald, MD

  ICM Co. Ltd.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: * Group 1: ICM-203 6x10e12 vg or Placebo * Group 2: ICM-203 2x10e13 vg or Placebo * Group 3: ICM-203 6x10e13 vg or Placebo (Optional)

Study Record Dates

• First Submitted: April 26, 2021
• First Posted: May 6, 2021
• Study Start: March 17, 2022
• Primary Completion: March 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: May 1, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (2)